Summary
- 2023 HDSA Annual Convention content now available online
- NIH Awards $22million to study of gene therapy for HD and related disorders
- FDA Grants Approval to Alzheimer’s drug, Leqembi
2023 HDSA Annual Convention content now available online
Whether you were unable to join us for this year’s Annual Convention or want to brush up on what you learned, HDSA has you covered! Recordings of all sessions from the 39th Annual HDSA Convention that took place in New Orleans, LA are now available on HDSA’s YouTube channel. Research highlights include the Clinical Trials Showcase, an update on the state of HD Research from the editors of HD Buzz, HD Research Participation 101, and more! Check out the full playlist here.
NIH Awards $22million to study of gene therapy for HD and related disorders
The NIH recently launched a new grant initiative to incentivize the development of gene-editing techniques that may be used to treat neurodegenerative diseases. Last week, it was announced that a group of researchers from UC Berkley, The Ohio State University, and UC San Francisco, will be awarded $22 million for the study of repeat expansion disorders, with a proposal that gives special priority to Huntington’s disease. The researchers’ goal is to use CRISPR technology to develop an investigative gene therapy that will selectively de-activate mutant copies of the huntingtin gene. If successful, this technology may have applications to other repeat expansion disorders as well, like ALS. The scientists, led by CRISPR gene-editing pioneer and Nobel Prize Winner, Dr. Jennifer Doudna, and primary investigator and clinician scientist, Dr. Krystof Bankiewicz, will aim to develop a drug that has the potential to reach clinical trials in five years’ time. It’s always wise to take such timelines with a grain of salt, as research can be unpredictable, but it’s exciting to hear about this large investment in genetic technologies for rare diseases from the US government. To read more, click here.
FDA Grants Approval to Alzheimer’s drug, Leqembi
Last week, the FDA granted full approval to leqembi, the first drug that has been shown to slow down symptoms of Alzheimer’s disease (AD). While leqembi does not stop or reverse AD, it has been observed to delay the progression of disease in certain individuals. The drug works by reducing the buildup of beta-amyloid plaques, the toxic protein clumps that accumulate in the brains of people with AD. The approval of leqembi means it will be widely covered by Medicare/Medicaid. While the drug will still come with a high price tag, this means it will be more accessible and affordable to people with AD than Aduhelm, the controversial drug that received conditional approval from the FDA in 2021. This victory for the Alzheimer’s community has many complexities, but there are lessons to be learned towards advocacy for rarer neurological diseases like HD. To read more, click here.
